Impact Biomedicines, Inc., a wholly owned subsidiary of Celgene Corporation
Individual Patient Compassionate Use of Fedratinib
Conditions: Myelofibrosis
This is an expanded access program (EAP) for eligible participants designed to provideaccess to fedratinib.Expanded access is only available in markets where fedratinib is not yet approved.
Gradalis, Inc.
Expanded Access of Vigil in Solid Tumors
Conditions: Solid Tumor, Ewing Sarcoma, Ewing's Tumor Metastatic, Ewing's Sarcoma Metastatic, Advanced Gynecological Cancers, Ovarian Cancer, Cervical Cancer, Uterine Cancer
This is an expanded access study involving an investigational product named Vigil. Vigilis considered immunotherapy. Patients who participated in another clinical trialsponsored by Gradalis, and had Vigil made from their tumor tissue removed from a standardoperation, however failed the criteria to enroll in the other clinical trial to receiveVigil are eligible to screen for this expanded access trial to receive the Vigil madefrom their cancer cells.In this study, eligible participants will receive intradermal (under the skin) injectionsof Vigil, once every 4 weeks (28 days) for 1-12 doses, depending on the number of dosesthat was made from the cancer cells and if the participant is clinically stable. Duringthe treatment portion of the study, in addition to receiving Vigil injections,participants will also have a physical exam, blood collection for routine and researchtests, and assessment of medications, adverse events, and performance status informationwill be collected. Radiological tumor assessments will be performed every 3 months fromCycle 1. Once treatment ends, participants will continue to be seen in the clinic every 3months for similar assessments until disease progression occurs. After diseaseprogression, participants will be contacted by phone 4 times a year to determine poststudy treatment and survival status information.
University of Texas, Southwestern Medical Center at Dallas
Intermediate Expanded Access Protocol CNMAu8.EAP03
Conditions: Multiple Sclerosis
This is a single-center intermediate expanded access program to provide access to theinvestigational product, CNM-Au8, up to 25 participants diagnosed with MultipleSclerosis.
Xcovery Holdings, Inc.
Expanded Access to Ensartinib for Participants With ALK+ NSCLC
Conditions: Non-Small Cell Lung Cancer, ALK Gene Rearrangement Positive
This is an open-label, multicenter, intermediate-sized expanded access treatment protocolto the existing IND 111,695 for ensartinib (X-396). The treatment plan is designed toprovide ensartinib to participants with anaplastic lymphoma kinase (ALK)-rearrangednon-small cell lung cancer (NSCLC).
ZevraDenmark
Early Access Program With Arimoclomol in US Patients With NPC
Conditions: Niemann-Pick Disease, Type C
NPC is a rare, relentlessly progressive, neurological disease and associated with seriousmorbidity and shortened life expectancy.The purpose of this Expanded Access Program is to provide early access to arimoclomol forpatients with Niemann-Pick Type C disease who, in the opinion and the clinical judgementof the treating physician, may benefit from treatment with arimoclomol.Participants will receive treatment with arimoclomol until their doctor finds it does nothelp them anymore, they withdraw, or the study is stopped for any reason.
Amicus Therapeutics
Expanded Access for ATB200/AT2221 for the Treatment of IOPD
Conditions: Pompe Disease Infantile-Onset
This is an expanded access program (EAP) for eligible participants designed to provideaccess to ATB200/AT2221.
Matrix Biomed, Inc.
A Single Patient Compassionate Use of MBM-02 (Tempol) for the Treatment of Prostate Cancer
Conditions: Prostate Cancer
A single patient compassionate use clinical trial for the use of MBM-02 (TEMPOL) for thetreatment of prostate cancer. The single patient will be exposed to orally administeredTEMPOL for up to 12 months at a maximum total daily dose of 800mg.